IRSF announces new research grants for translational research program and clinical trials

The International Rett Syndrome Foundation (IRSF) announces today ten (10) new research grants at leading global research institutions; a new Translational Research Program; and additional funding for current human clinical trials.

The funding awarded in 2013 covers the spectrum of Rett syndrome research, from basic discovery starting at the genetic level all the way to treating the girls diagnosed with Rett syndrome. Awards are not restricted by geographic boundaries, and several of the new IRSF grants are invested in researchers from Canada and Australia. IRSF remains steadfast in recruiting talented young scientists to the research bench for Rett syndrome, and welcomes three new IRSF fellows into the IRSF training fellowship program.

In an effort to move potential therapeutics past the translational research funding gap and into clinics, IRSF announces the Scout Program, a Drug Discovery Screen in a mouse model of Rett syndrome that will aggressively accelerate the testing of compounds, whether new or repurposed, through standardized preclinical studies. The Scout Program was created to replicate and confirm encouraging results stemming from studies performed in academic labs as well as testing of compounds no labs are pursuing. The Contract Research Organization (CRO) PsychoGenics, Inc. in Tarrytown, NY is the testing bed for this program and will independently test selected drugs using the set standards in preclinical studies for Rett syndrome. Outcomes from this program will provide the complementary high quality data required to move promising compounds through federal review and along the drug development pipeline toward the clinic.

"IRSF is taking the next logical step forward in developing programs that move the basic science discovery findings forward to rigorist pre-clinical trials. This is in concert with recent announcements and publications coming out of the National Institutes of Health for more rigor and replication of reported science before moving forward to clinical trials," said Steven Kaminsky, PhD, the Chief Science Officer of IRSF.

Rajat Shah, Chairman of the IRSF Board of Directors adds, "The Board is excited about the research direction that IRSF is taking. Accelerating the pace at which new drugs will move towards human clinical trials while identifying areas of research that need to be filled is of utmost importance to IRSF. Our sincerest thanks to all our families, donors, and supporters who have helped us fund this compelling research."

New Translational Research Awards

• Theresa E. Bartolotta, PhD, Seton Hall University and Patricia Remshifski, PhD, Monmouth University

• Eyetracking in Rett syndrome: A preliminary investigation of receptive and expressive vocabulary

• John Christodoulou, AM, Sydney Children's Hospital Network, Australia

• Preclinical Evaluation of Tubastatin A, a Novel Therapy for Rett Syndrome

• Steven Gray, PhD, University of North Carolina at Chapel Hill

• BDNF gene transfer for the treatment of RTT

• Jeannie Lee, MD, Ph.D, Massachusetts General Hospital

• Treating Rett syndrome via selective reactivation of the silenced MECP2 allele

New Basic Research Awards-Regular Research Grants

• Kerry Delaney, PhD, University of Victoria, Canada

• In vivo imaging of spine turnover rates in the heterozygous female Rett mouse brain

• Xinyu Zhao, PhD, University of Wisconsin-Madison

• Restoring network integration of MeCP2-deficient neurons

• Serena M. Dudek, PhD, National Institute of Environmental Health Sciences

• Role of IGF1R in hippocampal CA2 plasticity and function: interaction with MeCP2

New Mentored Training Fellowships

• Farshad Moradpour, PhD, University of Victoria, Canada

• Neurophysiology of cortical neurons in heterozygous female Rett mouse brain

• Deivid Rodrigues, PhD, The Hospital For Sick Children, Canada

• Post-transcriptional control of MECP2 expression during human development and disease

• Rocco George Gogliotti, PhD, Vanderbilt University Medical Center

• Temporal divergence of hypoconnectivity and excitotoxicity in Rett syndrome

New IRSF Scout Program

• Daniela Brunner, PhD, PyschoGenics, Inc.

• A Drug Discovery Screen in a Mouse Model of Rett Syndrome

Supplemental Funding for Current ANGEL Awards

• Walter Kaufmann, MD, Boston Children's Hospital

A Phase 2b placebo-controlled cross-over study of rh-IGF1 (mecasermin [DNA] injection) for treatment of Rett syndrome and development of Rett-specific novel biomarkers of cortical and autonomic function

• Daniel Glaze, MD, Baylor College of Medicine

A randomized, double-blind placebo controlled trial of NNZ-2566 (IGF-1{1-3}, glycyl-L-2-methylprolyl-L-glutamine acid) with open label extension in adults with Rett syndrome